Results 121 to 130 of about 423,061 (280)

SNUPN‐Related Muscular Dystrophy: Novel Phenotypic, Pathological and Functional Protein Insights

Annals of Clinical and Translational Neurology, EarlyView.
ABSTRACT Objective SNUPN‐related muscular dystrophy or LGMDR29 is a new entity that covers from a congenital or childhood onset pure muscular dystrophy to more complex phenotypes combining neurodevelopmental features, cataracts, or spinocerebellar ataxia. So far, 12 different variants have been described.
Nuria Muelas, Pablo Iruzubieta, Alberto Damborenea, Laura Pérez‐Fernández, Inmaculada Azorín, Juan Carlos Jiménez García, Ana Töpf, Pilar Martí, Lorena Fores‐Toribio, María Manterola, Rosana Blanco‐Mañez, Oihane Pikatza‐Menoio, Sonia Alonso‐Martín, Volker Straub, Aitziber L. Cortajarena, Adolfo López de Munain, David De Sancho, Lorea Blázquez, Juan J. Vilchez   +18 more
wiley   +1 more source

Monocyte LOXHD1 and RHOB Expression Predictive of Progressive Systemic Sclerosis–Associated Interstitial Lung Disease

Arthritis Care &Research, EarlyView.
Objective A leading cause of death among patients with scleroderma (SSc), interstitial lung disease (ILD) remains challenging to prognosticate. The discovery of biomarkers that accurately determine which patients would benefit from close monitoring and aggressive therapy would be an essential clinical tool.
Cristina M. Padilla, Robert Lafyatis, Kevin F. Gibson, Christina Morse, Eleanor Valenzi, Xiaoping Chen, Xiaoyun Li, Yanwu Zhao, Yongseok Park, Dana Ascherman, Jonathan Minden, Robyn Domsic, Yingze Zhang, Daniel J. Kass   +13 more
wiley   +1 more source

Stability of a stochastically perturbed model of intracellular single-stranded RNA virus replication

, 2019
Replication of single-stranded RNA virus can be complicated, compared to that of double-stranded virus, as it require production of intermediate antigenomic strands that then serve as template for the genomic-sense strands.
Elena, Santiago F., Korobeinikov, Andrei, Shaikhet, Leonid   +2 more
core   +1 more source

COVID‐19 Vaccination is not Associated with the Development of Idiopathic Inflammatory Myositis in U.S. Veterans

Arthritis Care &Research, Accepted Article.
Objective Several case reports have proposed a potential association between COVID‐19 vaccination and the subsequent development of idiopathic inflammatory myositis (IIM). This study examined prior COVID‐19 vaccination in US Veterans who developed new‐onset IIM compared to those without new‐onset IIM.
Caleb Hernández, Naomi Schlesinger, Jorge Rojas, Jessica A. Walsh, Tawnie J Braaten, Gary A Kunkel, Makoto Jones, Brian C Sauer, Julio Facelli, Grant W. Cannon, Dorota Lebiedz‐Odrobina   +10 more
wiley   +1 more source

Lipid Nanoparticles for the Delivery of CRISPR/Cas9 Machinery to Enable Site‐Specific Integration of CFTR and Mutation‐Agnostic Disease Rescue

Advanced Functional Materials, EarlyView.
Lipid nanoparticles (LNPs) are optimized to co‐deliver Cas9‐encoding messenger RNA (mRNA), a single guide RNA (sgRNA) targeting the endogenous cystic fibrosis transmembrane conductance regulator (CFTR) gene, and homologous linear double‐stranded donor DNA (ldsDNA) templates encoding CFTR.
Ruth A. Foley, Paul G. Ayoub, Vrishti Sinha, Colin Juett, Alicia Sanoyca, Emily C. Duggan, Lindsay E. Lathrop, Priyanka Bhatt, Kevin Coote, Beate Illek, Brigitte N. Gomperts, Donald B. Kohn, Steven J. Jonas   +12 more
wiley   +1 more source

Targeted dsRNA-mediated suppression of Phytophthora infestans infection via Avr3a

Applied Biological Chemistry
Phytophthora infestans (P. infestans) is a highly destructive oomycete that causes the late blight in Solanaceous crops, such as potatoes and tomatoes, reducing crop yield. Although many pesticides are used to control P.
Hyeonmin Lee, Minsu Park, Yujin Kweon, Dowhan Lee, Chanseok Shin   +4 more
doaj   +1 more source

ATPase activity profiling of three human DExD/H-box RNA helicases

SLAS Discovery
Human DExD/H-box RNA helicases are ubiquitous molecular motors that unwind and rearrange RNA secondary structures in an ATP-dependent manner. These enzymes play essential roles in nearly all aspects of RNA metabolism. While their biological functions are
Fengling Li, U Hang Chan, Julia Garcia Perez, Hong Zeng, Irene Chau, Yanjun Li, Almagul Seitova, Levon Halabelian   +7 more
doaj   +1 more source

FeDSNP‐Pa Nanoassemblies: A Triple‐Action Therapeutic Strategy Targeting Oxidative Stress, Inflammation, and Pyroptosis for Retinal Ganglion Cell Protection in Glaucoma

Advanced Functional Materials, EarlyView.
FeDSNP‐Pa, a metallized nanoparticle loaded with sodium pyruvate (Pa), exerts triple therapeutic effects by scavenging reactive oxygen species (ROS), suppressing inflammatory responses, and inhibiting pyroptosis signaling pathways. This multifunctional neuroprotective strategy protecting retinal ganglion cells (RGCs) from elevated intraocular pressure ...
Yukun Wu, Xue Li, Tong Wu, Dan Ji, Yugang Bai, Xuezhi Zhou   +5 more
wiley   +1 more source

Synergistic Osteogenesis After Co‐Administration of cmRNAs Encoding BMP‐2 and BMP‐7 Utilizing a Transcript‐Activated Matrix

Advanced Functional Materials, EarlyView.
This study demonstrates that the dual delivery of BMP‐2/‐7 coding cmRNAs for bone healing is demonstrated as feasible, safe, and highly osteogenic. Compared to single BMP‐2 or BMP‐7 cmRNAs, the combination enhances the production of both mineral and organic components of the extracellular matrix when delivered using a collagen‐HA scaffold, supporting ...
Claudia Del Toro Runzer, Joanna M. Sadowska, Nadia Roumans, Christian Plank, Christopher H. Evans, Fergal J. O'Brien, Martijn van Griensven, Elizabeth R. Balmayor   +7 more
wiley   +1 more source

Geminivirus sequences as bidirectional transcription termination/polyadenylation signals for economic construction of stably expressed transgenes [PDF]

, 2018
Bidirectional, convergent transcription of transgenes in transgenic plants can occur due to leaky transcription termination of separate convergent genes or from genomic promoters. It might also be engineered with the purpose of generating double-stranded
Bieri, Stéphane, Fütterer, Johannes, Potrykus, Ingo   +2 more
core